Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

Neuronal Ceroid Lipofuscinosis Clinical Trial

Official title:

A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT01238315
• Other study ID #: CL-N03-NCL
• Status: Withdrawn
• Phase: Phase 1
• First received: November 5, 2010
• Last updated: January 13, 2015
• Start date: November 2010
• Est. completion date: April 2011

Study information

• Verified date: January 2015
• Source: StemCells, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

The purpose of this Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression.

Description:

Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress immune system and a series of follow-up assessments.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: April 2011
• Est. primary completion date: April 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Months to 6 Years

Eligibility

Inclusion Criteria:

• Age 6 months to 6 years

• Male or female

• Clinical diagnosis of Infantile neuronal ceroid lipofuscinosis or late infantile neuronal ceroid lipofuscinosis

• CLN1 or CLN2 mutation

Exclusion Criteria:

• Previously received an organ, tissue or bone marrow transplantation

• Previously participated in any gene or cell therapy study

• Infection with hepatitis virus, cytomegalovirus, Epstein-Barr virus, or Human Immunodeficiency Virus (HIV)

• Current or prior cancer

• Bleeding disorder

• Unable to have an MRI scan

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Neuronal Ceroid Lipofuscinosis
• Neuronal Ceroid-Lipofuscinoses

Intervention

Biological:
• HuCNS-SC
Surgery to implant human CNS stem cells

Locations

Country	Name	City	State
United States	Oregon Health and Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
StemCells, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	number of participants with adverse events.	Safety will be measured by number of adverse events and changes in neurological status.	one year following transplantation	Yes
Secondary	Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS		one year following transplantation	No

External Links

•   Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis
•   Related Info